Treatment Trials

2 Clinical Trials for Various Conditions

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      NO_LONGER_AVAILABLE
      Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
      Description

      Expanded access to DCA as continued treatment for congenital lactic acidosis.

      Conditions
      Mitochondrial Enzyme Deficiencies
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      RECRUITING
      MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
      Description

      This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

      Conditions
      Mucopolysaccharidosis DisordersHurler SyndromeHunter SyndromeMaroteaux Lamy SyndromeSly SyndromeAlpha-MannosidosisFucosidosisAspartylglucosaminuriaGlycoprotein Metabolic DisordersSphingolipidosesRecessive LeukodystrophiesGloboid Cell LeukodystrophyMetachromatic LeukodystrophyNiemann-Pick BNiemann-Pick C Subtype 2Sphingomyelin DeficiencyPeroxisomal DisordersAdrenoleukodystrophy With Cerebral InvolvementZellweger SyndromeNeonatal AdrenoleukodystrophyInfantile Refsum DiseaseAcyl-CoA Oxidase DeficiencyD-Bifunctional Enzyme DeficiencyMultifunctional Enzyme DeficiencyAlpha-methylacyl-CoA Racmase DeficiencyMitochondrial Neurogastrointestingal EncephalopathySevere OsteopetrosisHereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)Inherited Metabolic Disorders
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